AVA-311 is being developed for the treatment of Juvenile X-linked Retinoschisis (XLRS), an inherited retinal disease that occurs almost exclusively in males caused by mutations in the RS1gene located on the X chromosome. AVA-311 is comprised of an optimized AAV vector using Adverum’s directed evolution technology that intravitreally delivers the RS1 gene in the 83 eye to potentially achieve a functional cure for patients.

In May 2014, Adverum signed a collaboration agreement with Regeneron for the development of AVA-311.

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